Study of the Outcome of Pediatric Patients with Severe Aplastic Anemia Treated with Hematopoietic Stem Cell Transplantation at Ain Shams University Pediatric Hospital

Abstract

Aplastic anemia (AA) is a syndrome of peripheral blood pancytopenia and bone marrow hypoplasia in the absence of major dysplasia, a malignant infiltrate or fibrosis. Clinical course of AA was found to be dependent on the degree of aplasia. In severe forms of the disease, rapid deterioration of patient's condition and possible incidence of fatal complications is the rule. HSCT from a matched sibling donor is the treatment of choice for children and young adults with severe aplastic anemia. However, it has several acute and chronic complications. All long- term survivors were subjected to the following assessment: Thorough history and clinical examination with detailed assessment of growth and development, symptoms, and signs of chronic GvHD in their follow-up visits Investigations for the graft included CBC with differential, reticulocyte counts and chimerism. Evaluation of immune reconstitution (IR) was done by flow-cytometry for CD markers (4, 8, 19 & 56) and by quantification of serum total IgG, IgM, and IgA. The median duration of the study was 87 months (43-115 months). The overall survival was 73.3%. Event free survival was 66.7%. All mortality was within 2 months post-transplant. Blood counts were within normal limits. Chimerism was complete for all except one. Immune reconstitution was consistent. Growth assessment revealed Z score of weight, height, and body mass index 0(-1.8-1.6), -0.7 (-1.8-0.95) and 0.34 (-1.29-2). Abnormal thyroid function was found in 3 patients (27%). No documented high grade acute graft-versus-host disease or extensive chronic graft-versus-host disease. Cardiac, pulmonary, hepatic and renal functions showed no abnormality, and no malignant complications were reported. Our study is limited with the small number of patients underwent HSCT for aplastic anemia in our unit, but the long-term follow-up added to its strength. And further studies are still needed on bigger number of cases.